Abstract
Familial Danish dementia (FDD), also known as heredopathia ophthalmo-oto-encephalica, is an autosomal dominant disorder characterized by cataracts, deafness, progressive ataxia, and dementia. Neuropathological findings include severe widespread cerebral amyloid angiopathy, hippocampal plaques, and neurofibrillary tangles, similar to Alzheimer's disease. N-terminal sequence analysis of isolated leptomeningeal amyloid fibrils revealed homology to ABri, the peptide originated by a point mutation at the stop codon of gene BRI in familial British dementia. Molecular genetic analysis of the BRI gene in the Danish kindred showed a different defect, namely the presence of a 10-nt duplication (795-796insTTTAATTTGT) between codons 265 and 266, one codon before the normal stop codon 267. The decamer duplication mutation produces a frame-shift in the BRI sequence generating a larger-than-normal precursor protein, of which the amyloid subunit (designated ADan) comprises the last 34 C-terminal amino acids. This de novo-created amyloidogenic peptide, associated with a genetic defect in the Danish kindred, stresses the importance of amyloid formation as a causative factor in neurodegeneration and dementia.
MeSH Terms
3' Untranslated Regions/genetics
Adaptor Proteins, Signal Transducing
Amino Acid Sequence
Amyloid/analysis,genetics
Base Sequence
Dementia/genetics,pathology
Denmark
Female
Gene Duplication
Humans
Male
Membrane Glycoproteins
Membrane Proteins
Molecular Sequence Data
Pedigree
Sequence Alignment
Sequence Homology, Amino Acid
Temporal Lobe/blood supply,pathology
Whites
Chemicals
3' Untranslated Regions
Adaptor Proteins, Signal Transducing
Amyloid
ITM2B protein, human
Membrane Glycoproteins
Membrane Proteins
Authors & Affiliations
11 authors, click to expand affiliations / ORCID
Vidal R
Department of Pathology, New York University School of Medicine, New York 10016, USA. vidalr01@popmail.med.nyu.edu
Revesz T
Rostagno A
Kim E
Holton J L
Bek T
Bojsen-Møller M
Braendgaard H
Plant G
Ghiso J
Frangione B
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